Lentiviral-mediated Gene Therapy of Fanconi Anemia Patients Subtype A (FANCOLEN-1)
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|ClinicalTrials.gov Identifier: NCT03157804|
Recruitment Status : Active, not recruiting
First Posted : May 17, 2017
Last Update Posted : November 25, 2020
This is an open, Phase I / II clinical trial to evaluate the safety and efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi Anemia of Subtype A .
CD34 + cells derived from bone marrow and / or mobilized peripheral blood (fresh and / or cryopreserved) from patients with Fanconi subtype A (FA-A), will be transduced ex vivo with a lentiviral vector carrying the gene FANCA (orphan drug) . After transduction the cells will be inoculated in patients in order to restore their hematopoiesis with genetically corrected stem cells.
|Condition or disease||Intervention/treatment||Phase|
|Fanconi Anemia||Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs) Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells Other: Laboratory Biomarker Analysis Biological: Filgrastim Drug: Plerixafor Procedure: Bone Marrow Aspiration||Phase 1 Phase 2|
The main objective of this open-label Phase I / II clinical trial is to evaluate the safety and therapeutic efficacy of a hematopoietic gene therapy procedure with an orphan drug consisting of a lentiviral vector carrying the FANCA gene for patients with Fanconi's Anemia Subtype A.
The drug to be administered to the patients consists of the cellular product resulting from the transduction of autologous CD34 + cells with the therapeutic lentiviral vector PGK-FANCA.Wpre *.
The dose of cells to infuse in the patients will be that obtained from the transduction process of between 3x10^5 and 4x10^6 CD34 + cells / kg of patient body weight.
The cells will be infused intravenously in a single dose, after complete the transduction process.
Follow-up period: 3 years after infusion of transduced cells. However, patients will be monitored outside the clinical trial over a 10-year period.
Follow-up of the grafted transduced cells will be performed on peripheral blood and bone marrow samples.
|Study Type :||Interventional (Clinical Trial)|
|Actual Enrollment :||9 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||Clinical Trial Phase I / II to Evaluate the Safety and Efficacy of the Infusion of Autologous CD34 + Cells Transduced With a Lentiviral Vector Carrying the Gene FANCA in Patients With FA Subtype A (FANCOLEN-1)|
|Actual Study Start Date :||January 7, 2016|
|Actual Primary Completion Date :||April 23, 2019|
|Estimated Study Completion Date :||April 2022|
Experimental: Autologous CD34+ cells transducted with PGK-FANCA-Wpre *
CD34 + cells from patients with Fanconi subtype A (FA-A) transduced ex vivo with lentiviral vector carrying the gene FANCA, PGK-FANCA-Wpre*The product to be infused consist of a suspension of transduced CD34^+ cells.
Procedure: IV administration of Genetically Engineered Hematopoietic Stem/Progenitors Cells (HSPCs)
Biological: Genetically Engineered Hematopoietic Stem/Progenitor Cells
Undergo infusion of genetically modified hematopoietic progenitor cell therapy
Other Name: Genetically Engineered HSPCs
Other: Laboratory Biomarker Analysis
Given subcutaneously (SC)
Other Name: Filgrastim XM02, Filgrastim-sndz, G-CSF (Colony Stimulating Factor), Neupogen, r-metHug-CSF, Recombinant Methionyl Human Granulocyte CSF, rG-CSF, Tbo-filgrastim, Zarxio
Other Name: AMD 3100, JM-3100, Mozobil, SDZ SID 791
Procedure: Bone Marrow Aspiration
- Number of participants with treatment-related adverse events as assessed by CTCAE v4.0 [ Time Frame: Up to 3 years after infusion of transduced cells ]All adverse events will be registered for 3 years from infusion of transduced cells
- Proportion of patients with at least 0.1 copy of the therapeutic vector per nucleated bone marrow or peripheral blood cells three years after infusion. [ Time Frame: 3 years after infusion of transduced cells ]Detection of at least 0.1 copy of the therapeutic vector per nucleated bone marrow cell or peripheral blood cells three years after infusion.
- Proportion of patients with clinical hematological response after the infusion of autologous CD34 + cells transduced with the therapeutic lentiviral vector [ Time Frame: 3 years after infusion of transduced cells ]Proportion of patients with clinical hematological response (improvement of cell blood counts at least in one hematological lineage).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03157804
|Hospital Vall d'Hebron|
|Barcelona, Spain, 08035|
|Hospital Infantil del Niño Jesus|
|Madrid, Spain, 28009|
|Study Director:||Juan A Bueren||CIEMAT/CIBERER/IIS.FJD|